Blog
RSS FeedPosted by Michael Wonder on 11 Aug 2024
Satellos receives rare paediatric disease designation from the US FDA for SAT-3247 for the treatment of Duchenne muscular dystrophy
8 August 2024 - Satellos Bioscience announced today that the US FDA has granted rare paediatric disease designation to SAT-3247 ...
Posted by Michael Wonder on 07 Aug 2024
Meta Pharmaceuticals announces FDA grants rare paediatric disease designation to META-001-PH for the treatment of primary hyperoxaluria
5 August 2024 - META Pharmaceuticals Inc. announced that the US FDA has granted rare paediatric disease designation to its investigational ...
Posted by Michael Wonder on 05 Aug 2024
FDA submission to expand minimum age for Xenoview
1 August 2024 - Polarean Imaging announces that it has submitted a new drug application supplement to the US FDA to ...
Posted by Michael Wonder on 03 Aug 2024
Drugs in rare paediatric disease priority review voucher program have similar revenue to brand drugs
2 August 2024 - Drugs issued rare paediatric disease priority review vouchers that were redeemed had revenues that were similar ...
Posted by Michael Wonder on 26 Jul 2024
US FDA approves BioMarin's Brineura (cerliponase alfa) for children under 3 years with CLN2 disease
24 July 2024 - Now approved for children of all ages with CLN2 Batten disease, regardless of whether they yet ...
Posted by Michael Wonder on 25 Jul 2024
ConSynance Therapeutics receives FDA rare paediatric disease designation for CSTI-500 in Prader-Willi syndrome
24 July 2024 - ConSynance Therapeutics today announced that the US FDA has granted rare paediatric disease designation to its ...
Posted by Michael Wonder on 24 Jul 2024
FDA accepts Mesoblast's biologics license application for Ryoncil in children with steroid-refractory acute graft versus host disease
23 July 2024 - Mesoblast announced that the US FDA has accepted its biologics license application resubmission for Ryoncil (remestemcel-L) ...
Posted by Michael Wonder on 20 Jul 2024
Neuren receives rare paediatric disease designation from FDA
19 July 2024 - Neuren Pharmaceuticals has received rare paediatric disease designation from the US FDA for NNZ-2591 in Phelan-McDermid ...
Posted by Michael Wonder on 18 Jul 2024
Sellas announces US FDA rare paediatric disease designation granted to SLS009 for the treatment of paediatric acute myeloid leukaemia
16 July 2024 - Opened enrollment for paediatric AML patients in on-going Phase 2 clinical trial. ...
Posted by Michael Wonder on 13 Jul 2024
Innorna announces US FDA rare paediatric disease designation granted to IN016 for the treatment of progressive familial intrahepatic cholestasis
11 July 2024 - Innorna today announced that the US FDA has granted rare paediatric disease designation to IN016, one of ...
Posted by Michael Wonder on 03 Jul 2024
The rare paediatric disease voucher program creates new treatments. I have new data to prove it.
2 July 2024 - The FDA’s rare paediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations ...
Posted by Michael Wonder on 26 Jun 2024
Harmony Biosciences receives US FDA approval for Wakix (pitolisant) in paediatric patients with narcolepsy
24 June 2024 - FDA granted priority review of the sNDA in February 2024. ...
Posted by Michael Wonder on 25 Jun 2024
Sellas announces US FDA rare paediatric disease designation granted to SLS009 for the treatment of paediatric acute lymphoblastic leukaemia
24 June 2024 - Sellas Life Sciences today announced that the US FDA has granted rare paediatric disease designation to SLS009, ...
Posted by Michael Wonder on 18 Jun 2024
US FDA rare paediatric disease designation granted to RC220 bisantrene for the treatment of paediatric AML
18 June 2024 - Race Oncology is pleased to announce that the US FDA has extended rare paediatric disease designation ...
Posted by Michael Wonder on 12 Jun 2024
Farxiga approved in the US for the treatment of paediatric type 2 diabetes mellitus
12 June 2024 - Approval based on results from T2NOW, one of the largest paediatric type 2 diabetes Phase 3 ...